ROCKVILLE, Md., Oct. 11, 2019 /PRNewswire/ -- REGENXBIO Inc.
(Nasdaq: RGNX), a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene
therapy based on its proprietary NAV® Technology
Platform, today announced interim data from the ongoing Phase I/IIa
trial of RGX-314 for the treatment of wet age-related macular
degeneration (wet AMD). The results were presented by Jeffrey S. Heier, M.D., Co-President and
Director of Retina Research at Ophthalmic Consultants of
Boston and primary investigator
for the trial, in a podium presentation at the Retina Subspecialty
Day program of the American Academy of Ophthalmology (AAO) 2019
Annual Meeting in San Francisco,
CA.
"Today's interim update from the RGX-314 Phase I/IIa dose
escalation study further demonstrates the significant reduction in
anti-VEGF treatment burden and encouraging improvement or
maintenance of effects on vision and retinal thickness in the three
higher dose cohorts," said Dr. Heier. "These effects are especially
important as subjects in this study had been previously treated
with chronic and burdensome anti-VEGF injections over several
years, highlighting the severity of their disease. Today's results
further support the potential of RGX-314 gene therapy to have
meaningful and durable effects in patients following a one-time
intervention."
Detailed study findings, including those presented by Dr. Heier
at AAO 2019, are available under the Presentations &
Publications page in the Media section of the company's website
located at www.regenxbio.com.
Study Design and Safety
In the Phase I/IIa trial of RGX-314, 42 subjects with severe wet
AMD requiring frequent anti-vascular endothelial growth factor
(anti-VEGF) injections have been treated across five dose cohorts,
with doses ranging from 3x109 GC/eye to
2.5x1011 GC/eye. Subjects were enrolled into all dose
cohorts independent of their neutralizing antibody titers to AAV
and did not receive prophylactic immune suppressive oral
corticosteroid therapy before or after administration of
RGX-314.
Subjects in the study are being assessed each month, with
long-term follow-up continuing for 24 months. Assessments for the
study include reduction in anti-VEGF intravitreal injections,
change in vision measured by Best Corrected Visual Acuity (BCVA),
change in central retinal thickness (CRT) measured by spectral
domain optical coherence tomography (SD-OCT), and protein
expression levels as measured from aqueous samples by
electrochemiluminescence immunoassay (ECL).
As of October 9, 2019, RGX-314
continues to be well-tolerated across all cohorts, with no
drug-related serious adverse events (SAEs) reported. Fifteen SAEs
that were not related to RGX-314, including two ocular
procedure-related SAEs, were reported in 9 subjects. There have
been no reports of clinically-determined immune responses,
drug-related ocular inflammation, or post-surgical inflammation
beyond what is expected following routine vitrectomy.
Summary of Data for Cohorts 4 and 5
Today's interim update includes data as of October 9, 2019 for Cohorts 4 and 5, which
enrolled 12 subjects each, at doses of 1.6x1011 GC/eye
and 2.5x1011 GC/eye, respectively. All 12 subjects in
Cohort 4 reached 6 months of follow-up, and subjects in Cohort 5
reached 5 or 6 months of follow-up as of the data cut-off, with the
exception of one subject who discontinued from the study at 4
months.1
Subjects in Cohort 5 on average had a meaningful reduction in
anti-VEGF treatment burden, with 9 out of 12 (75%) subjects
remaining anti-VEGF injection-free as of the data cut-off. Across
the 12 subjects, there was a mean of 0.8 injections through 5 or 6
months following administration of RGX-314, a reduction of over 80%
from the mean annualized injection rate during the 12 months prior
to administration of RGX-314. Importantly, subjects in Cohort 5
improved visual acuity and decreased retinal thickness, with a mean
BCVA change of +4 letters and a mean change in CRT of -68µm after
one-time administration of RGX-314. The 9 subjects who were
anti-VEGF injection-free after administration of RGX-314 showed a
mean BCVA improvement of +5 letters, and a mean improvement in CRT
of -80µm.
Subjects in Cohort 4 on average also had a meaningful reduction
in anti-VEGF treatment burden, with 5 out of 12 (42%) subjects
receiving no anti-VEGF injections in 6 months following
administration of RGX-314. Across the 12 subjects in the cohort,
there was a mean of 2.2 injections over 6 months following
administration of RGX-314, a reduction of over 50% from the mean
annualized injection rate during the 12 months prior to
administration of RGX-314. Subjects in Cohort 4 maintained visual
acuity and decreased retinal thickness, with a mean BCVA change of
+2 letters, and a mean change in CRT of -42 µm. The 5 subjects who
did not receive anti-VEGF injections after administration of
RGX-314 showed a mean BCVA change of +2 letters, and a mean
improvement in CRT of -61µm.
Intraocular RGX-314 protein expression levels increased in a
dose-dependent manner when measured at approximately one month
after administration of RGX-314; the mean protein expression level
in Cohort 4 was 249.4 ng/ml, and the mean protein expression level
in Cohort 5 was 376.0 ng/ml.
Summary of Long-Term Data for Cohort 3
Subjects in Cohort 3 continue to demonstrate long-term
reductions in anti-VEGF treatment burden over 1.5 years.
Importantly, 3 out of 6 subjects (50%) continue to remain anti-VEGF
injection-free at 1.5 years. The 6 subjects across the cohort
demonstrated a mean annualized rate of 2.6 anti-VEGF injections
following administration of RGX-314, a reduction of over 50% from
the mean annualized injection rate during the 12 months prior
administration of RGX-314.
Positive long-term efficacy signals were sustained through 1.5
years in Cohort 3, including a mean BCVA improvement of +9 letters
and a mean change in CRT of -40 µm. Notably, in the three patients
who have remained anti-VEGF injection free at 1.5 years, the
increase from baseline BCVA was +11 letters and the mean change in
CRT was -21 µm.
"Frequent anti-VEGF injections have been shown to reduce the
risk of blindness in subjects with wet AMD, but real-world evidence
shows that people lose vision over time due to non-compliance. The
notable reduction in anti-VEGF treatments seen after a single
administration of the highest dose of RGX-314 in Cohort 5 is
particularly encouraging, given the severity of the disease and the
high treatment burden for these enrolled subjects prior to
administration of RGX-314," said Steve
Pakola, M.D., Chief Medical Officer of REGENXBIO. "We look
forward to our anticipated start of the Phase IIb trial in subjects
with wet AMD by the end of this year."
About RGX-314
RGX-314 is being developed as a potential one-time treatment for
wet age-related macular degeneration (wet AMD), diabetic
retinopathy (DR), and other additional chronic retinal conditions
treated with anti-VEGF. RGX-314 consists of the NAV AAV8 vector
encoding an antibody fragment which inhibits VEGF, modifying the
pathway for formation of new leaky blood vessels which lead to
retinal fluid accumulation and vision loss.
About the Phase I/IIa Clinical Trial of RGX-314
RGX‑314 is being evaluated in a Phase I/IIa, multi-center,
open-label, multiple-cohort, dose‑escalation study in adult
subjects with wet AMD in the United
States. The study includes subjects previously treated for
wet AMD who are responsive to anti-VEGF therapy. The study is
designed to evaluate five escalating doses of RGX-314, with six
subjects in the first three dose cohorts and 12 subjects in the
fourth and fifth dose cohorts. Subjects were enrolled into all dose
cohorts independent of their neutralizing antibody titers to AAV
and did not receive prophylactic immune suppressive oral
corticosteroid therapy before or after administration of RGX-314.
Secondary endpoints include visual acuity, retinal thickness on
spectral domain optical coherence tomography (SD‑OCT), ocular
RGX-314 protein expression, and the need for additional anti-VEGF
therapy. Following completion of the primary study period, subjects
enter a follow-up period and will continue to be assessed until
week 106 for long-term safety and durability of effect.
About Wet AMD
Wet AMD is characterized by loss of vision due to new, leaky
blood vessel formation in the retina. Wet AMD is a significant
cause of vision loss in the United
States, Europe and
Japan, with up to 2 million people
living with wet AMD in these geographies alone. Current anti-VEGF
therapies have significantly changed the landscape for treatment of
wet AMD, becoming the standard of care due to their ability to
prevent progression of vision loss in the majority of patients.
These therapies, however, require life-long intraocular injections,
typically repeated every four to 12 weeks in frequency, to maintain
efficacy. Due to the burden of treatment, patients often experience
a decline in vision with reduced frequency of treatment over
time.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene
therapy. REGENXBIO's NAV Technology Platform, a proprietary
adeno-associated virus (AAV) gene delivery platform, consists of
exclusive rights to more than 100 novel AAV vectors, including
AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV
Technology Platform Licensees are applying the NAV Technology
Platform in the development of a broad pipeline of candidates in
multiple therapeutic areas.
Forward-Looking Statements
This press release includes "forward-looking statements," within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. These statements express a belief, expectation or
intention and are generally accompanied by words that convey
projected future events or outcomes such as "believe," "may,"
"will," "estimate," "continue," "anticipate," "design," "intend,"
"expect," "could," "plan," "potential," "predict," "seek,"
"should," "would" or by variations of such words or by similar
expressions. The forward-looking statements include statements
relating to, among other things, REGENXBIO's future operations and
clinical trials. REGENXBIO has based these forward-looking
statements on its current expectations and assumptions and analyses
made by REGENXBIO in light of its experience and its perception of
historical trends, current conditions and expected future
developments, as well as other factors REGENXBIO believes are
appropriate under the circumstances. However, whether actual
results and developments will conform with REGENXBIO's expectations
and predictions is subject to a number of risks and uncertainties,
including the timing of enrollment, commencement and completion and
the success of clinical trials conducted by REGENXBIO, its
licensees and its partners, the timing of commencement and
completion and the success of preclinical studies conducted by
REGENXBIO and its development partners, the timely development and
launch of new products, the ability to obtain and maintain
regulatory approval of product candidates, the ability to obtain
and maintain intellectual property protection for product
candidates and technology, trends and challenges in the business
and markets in which REGENXBIO operates, the size and growth of
potential markets for product candidates and the ability to serve
those markets, the rate and degree of acceptance of product
candidates, and other factors, many of which are beyond the control
of REGENXBIO. Refer to the "Risk Factors" and "Management's
Discussion and Analysis of Financial Condition and Results of
Operations" sections of REGENXBIO's Annual Report on Form 10-K for
the year ended December 31, 2018, and
comparable "risk factors" sections of REGENXBIO's Quarterly Reports
on Form 10-Q and other filings, which have been filed with the U.S.
Securities and Exchange Commission (SEC) and are available on the
SEC's website at www.sec.gov. All of the forward-looking statements
made in this press release are expressly qualified by the
cautionary statements contained or referred to herein. The actual
results or developments anticipated may not be realized or, even if
substantially realized, they may not have the expected consequences
to or effects on REGENXBIO or its businesses or operations. Such
statements are not guarantees of future performance and actual
results or developments may differ materially from those projected
in the forward-looking statements. Readers are cautioned not to
rely too heavily on the forward-looking statements contained in
this press release. These forward-looking statements speak only as
of the date of this press release. REGENXBIO does not undertake any
obligation, and specifically declines any obligation, to update or
revise any forward-looking statements, whether as a result of new
information, future events or otherwise.
1 This subject died 4.5 months after the
administration of RGX-314 as a result of the subject's underlying
disease, which was assessed to be unrelated to RGX-314. At the time
of the death, the subject was free of anti-VEGF injections.
Contacts:
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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